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Treatment of Neuronopathic Mucopolysaccharidoses with Blood–Brain Barrier-Crossing Enzymes: Clinical Application of Receptor-Mediated Transcytosis

Enzyme replacement therapy (ERT) has paved the way for treating the somatic symptoms of lysosomal storage diseases (LSDs), but the inability of intravenously administered enzymes to cross the blood–brain barrier (BBB) has left the central nervous system (CNS)-related symptoms of LSDs largely impervi...

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Detalles Bibliográficos
Autores principales: Sonoda, Hiroyuki, Takahashi, Kenichi, Minami, Kohtaro, Hirato, Toru, Yamamoto, Tatsuyoshi, So, Sairei, Tanizawa, Kazunori, Schmidt, Mathias, Sato, Yuji
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9229961/
https://www.ncbi.nlm.nih.gov/pubmed/35745811
http://dx.doi.org/10.3390/pharmaceutics14061240